On May 2, 2018 AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, reported financial results for the first-quarter ended March 31, 2018 (Press release, AC Immune, MAY 2, 2018, View Source [SID1234525983]).

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Prof. Andrea Pfeifer, CEO of AC Immune, commented: "During the first quarter we announced plans to start the first in human study of potentially the first selective alpha-synuclein PET tracer for earlier and more accurate diagnosis of Parkinson’s disease. Furthermore, our Tau small molecules (Tau Morphomers) demonstrated target-specific reduction of pathological Tau as well as cognitive and functional improvement. We also presented a number of scientific updates on our programs at a prestigious scientific conference, demonstrating progress in our key areas. The Company’s financial position remains strong, while we continue to invest in our highly-valued R&D resources as we pursue our mission of being a leader in precision medicine for neurodegenerative diseases.

First Quarter 2018 Company Highlights

First potential PET tracer for Parkinson’s disease
We announced the first in human study for potentially the first alpha-synuclein positron emission tomography (PET) tracer for earlier and more accurate diagnosis of Parkinson’s disease. This new compound is highly selective for alpha-synuclein aggregates, a recognized and known target for Parkinson’s disease and diseases involving alpha-synuclein pathologies. Data was presented at the AAT-AD/PD Focus Meeting 2018 in Torino, Italy on March 15, 2018.

Scientific updates at AAT- AD/PD Focus Meeting
The Company and its partners provided updates at the AAT-AD/PD Focus Meeting in Torino, Italy, in March 2018: Several posters and oral presentations covered the alpha-synuclein PET tracer being developed together with Biogen; the Company’s in-house SupraAntigenTM vaccine technology; the anti-amyloid-ß antibody crenezumab currently in Phase 3 development with our collaboration partner Genentech, a member of the Roche Group; and the Tau-PET imaging agent PI-2620 being developed with Piramal Imaging.

Selection of Tau Small Molecules for Clinical Development in Alzheimer’s disease
AC Immune has one of the largest Tau pipeline in the industry. Several Tau small molecule candidates, derived from AC Immune’s proprietary MorphomerTM platform and designed to cross the blood brain barrier, have demonstrated target-specific reduction of pathological Tau and cognitive and functional improvement in proof-of-concept studies in Alzheimer’s disease. IND/CTA enabling studies have started and a Phase 1 study will commence by the end of 2018.

Continue to invest in our key R&D programs and resources
During the quarter we added 7.3 FTEs to our R&D operations, as we continue to invest in our non-Alzheimer’s disease research, diagnostics and new discovery programs as we seek to position the Company clearly as a leader in precision medicine for neurodegenerative diseases.

First Quarter 2018 Financial Highlights

Revenues
Our revenues fluctuate as a result of our collaborations with current and potentially new partners, the timing of milestone achievements, and the size of each milestone payment.
AC Immune generated revenues of CHF 1.5 million in the three months ended March 31, 2018, a decrease of CHF 0.5 million over the comparable period in 2017. The decrease in collaboration revenues was principally due to the recognition of a EUR 1 million (CHF 1.1 million) milestone from Piramal for the initiation of the Phase 1 clinical trial in an orphan indication, Progressive Supranuclear Palsy (PSP), which did not reoccur in Q1 2018.

Research & Development (R&D) Expenses
In the first quarter of 2018, AC Immune invested CHF 10.1 million in research and development, compared with CHF 7.5 million for the same period in 2017. The increase in R&D programs is primarily driven by increased investments in our two ACI 24 programs in Alzheimer’s disease (AD) and Down syndrome, our Anti-Tau vaccines for the treatment of AD, our Anti-Tau Morphomers small molecules and alpha-synuclein Antibody program.

General and Administrative (G&A) Expenses
General and administrative expenses amounted to CHF 2.7 million in the three months ended March 31, 2018, compared with CHF 2.4 million in the same period in 2017.

IFRS Loss for the period
For the three months ended March 31, 2018, the Company had a net loss of CHF 11.6 million compared with net loss of CHF 9.4 million for the same period in 2017. The decline in profitability is attributable to the decreased revenues for the periods as a result of prior milestone achievements and an increase in R&D and G&A expenses as outlined above.

Cash position
As of March 31, 2018, AC Immune had total cash of CHF 109.7 million compared to CHF 124.4 million as of December 31, 2017. The decrease of CHF 14.7 million is principally due to the net loss of CHF 11.6 million for the three month period. Net cash flows used in operating activities were CHF 13.3 million, due to the higher investments in our major discovery and development programs, and the continued strengthening of the Company’s infrastructure, systems and organization as a publicly-traded company.

Non-IFRS Financial Measures
In addition to our operating results, as calculated in accordance with International Financial Reporting Standards, or IFRS, as adopted by the International Accounting Standards Board, we use Adjusted Loss and Adjusted Loss per Share when monitoring and evaluating our operational performance. Adjusted Loss is defined as loss for the relevant period, as adjusted for certain items that we believe are not indicative of our ongoing operating performance. Adjusted Loss per Share is defined as Adjusted Loss for the relevant period divided by the weighted-average number of shares for such period.

1 Reflects non-cash expenses associated with share-based compensation for equity awards issued to Directors, Management and employees of the Company. This expense reflects the awards’ fair value recognized for the portion of the equity award which is vesting over the period.

2 Reflects foreign currency remeasurement gains and losses for the period, predominantly impacted by the change in the exchange rate between the US Dollar and the Swiss Franc.

Non-IFRS Expenditures
Adjustments for the three ended March 31, 2018 and 2017 were CHF 0.8 million and CHF 1.7 million in net losses, respectively. These were largely due to foreign currency remeasurement losses of CHF 0.2 million and CHF 1.6 million, respectively, predominantly related to the cash balance of the Company as a result of a weakening of the US Dollar against the Swiss Franc for most of the first half of the year offset by gains in the third quarter.
The Company also recorded CHF 0.6 million and CHF 0.1 million for share-based compensation expenses during the three months ended March 31, 2018 and 2017, respectively

On May 2, 2018 IONTAS and aTen Therapeutics announce collaboration to discover therapeutic oncology leads Cambridge, UK and Edinburgh, UK, 02 May 2018: IONTAS Limited (IONTAS), a leader in the discovery and optimisation of fully human antibodies, and aTen Therapeutics Limited (aTen), a private biopharmaceutical company developing next-generation antibodies for the treatment of cancer, reported that they have entered into a strategic collaboration (Press release, Iontas, MAY 2, 2018, View Source [SID1234525982]). Under the agreement IONTAS will use its proprietary antibody discovery platforms to deliver antibodies against biological targets selected by aTen.

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Dr John McCafferty, CEO and Founder of IONTAS, said: "The use of traditional in vivo technologies can work well to generate simple therapeutic antibodies, but with increasing requirements for specificity, function, developability, and affinity, these traditional approaches are not always appropriate. IONTAS will apply its technologies to overcome limitations of other platforms to generate therapeutic leads against pre-defined specification from aTen, to help its current pipeline progress."

Prof Chris Wood, Chairman and Founder of aTen, commented: "IONTAS was selected because of its technological expertise and proven track record of delivering antibodies to all its partners. We value the extensive experience IONTAS can add to advancing aTen’s therapeutic programs in the fight against cancer."

Dr John McCafferty will present "Antibody-display libraries in mammalian cells created using CRISPR/cas9 and TALE nuclear essence" at 11:25 EST on 02 May 2018 during the Therapeutics & Technologies Stream – CRISPR FOR GENOME ENGINEERING at PEGS in Boston, MA.

For further information, please visit View Source

On May 2, 2018 Refuge Biotechnologies, Inc. ("Refuge"), a company leveraging gene engineering technologies to develop intelligent cell therapeutics programmed to make decisions inside of patients, reported the closing of a $25 million Series B investment round (Press release, refuge biotechnologies, MAY 2, 2018, View Source [SID1234525981]). In addition, the company has appointed immuno-oncology pioneer Francesco Marincola, M.D., as chief scientific officer.

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The Series B financing was led by 3SBio and Sequoia China, with participation from new investors Danhua Capital (DHVC), Sangel Capital and Ocean Pine Healthcare Fund. Refuge’s existing investors, 3E Bioventures, WuXi Healthcare Ventures, and ShangBay Capital, also participated in the round.

The funds from the Series B will support advancement of cell therapies developed with Refuge’s receptor-dCas platform, which utilizes a mutated or dead Cas9 (dCas) as a targeting mechanism to enable precision CRISPR activation (CRISPRa) and CRISPR interference (CRISPRi). The cell therapies are programed to only activate CRISPRa/CRISPRi when they encounter specific sensors found on the surface of cancer cells, which delivers the treatment effect only to target cells. As a result, cell therapies have the potential to bring together multiple anti-cancer approaches in a single cell, such as repression of multiple checkpoint targets, with greater potency and reduced side effects. Refuge’s pipeline is led by RB-1916, a CAR-T cell therapy designed to inhibit the expression of the PD-1 gene, with a potential initial application in diffused large B-cell lymphoma. Refuge has additional CAR cell therapy programs under research that conditionally repress PD-1 and other checkpoint inhibitors for potential treatment of solid tumors.

"We have seen tremendous progress in the development of our technology and science, and believe that our receptor-dCas platform has the potential to create highly targeted cell therapies that bring superior efficacy while overcoming limitations related to toxic side effects," said Bing C. Wang, Ph.D., co-founder and chief executive officer of Refuge Biotech. "This financing will propel our efforts with our growing pipeline as we continue to design these innovative and intelligent cell therapies to fight cancer, and we are encouraged by the support from this top group of global investors."

As part of the investment, the lead investors will have an exclusive right to negotiate with Refuge on the right to the development and commercialization of cell therapies using Refuge’s platform in China. Concurrently, Refuge and 3SBio will also collaborate on research developing programmed cell therapeutics that can produce therapeutic proteins inside a patient’s body using Refuge’s platform technology.

Concurrent with the financing, Zhenping Zhu, M.D., Ph.D., of 3SBio and Trency Gu, Ph.D., of Sequoia China, have joined the Refuge board of directors.

"3SBio’s investment demonstrates our commitment to advancing cutting-edge gene engineering technologies with potential for breakthrough treatments for cancer and other diseases with unmet medical needs," said Jing Lou, M.D., Ph.D., Chairman and CEO of 3SBio Inc. "3SBio looks forward to collaborating with Refuge to accelerate the clinical development of Refuge’s next-generation cell therapies for cancer and to fully realize the potential of the dCas9 platform."

Added Neil Shen, founding and managing partner of Sequoia China, "Sequoia China endeavors to back innovative companies in the life science field such as Refuge Bio, which brings together topnortch scientific and commercial talents in the gene editing and cell therapy space. We are pleased to support Refuge Bio to further develop the dCas9 platform for wide therapeutic applications to improve human health."

The CRISPRa and CRISPRi are made possible by dCas9, which no longer cuts DNA but functions as a carrier to specific areas of the genome for highly targeted delivery a transcriptional activator or repressor to turn on or turn off genes. The novel receptor-dCas platform allows for control of how a cell interacts with its environment. Cells generally communicate and sense their surrounding through membrane receptors. Connecting receptors to dCas creates a therapeutic platform that enables cells to sense its surroundings and activate or repress multiple gene expression based on the receptor-ligand interactions. With receptor-dCas, cells can be now programmed to turn off certain genes, such as PD-1, to generate more potent CAR-T immune cells when it senses the presence of a tumor cell.

About Francesco Marincola, M.D.
As chief scientific officer, Dr. Marincola will lead development of Refuge’s intelligent cell therapy platform and investigation of its lead therapeutic programs. He most recently served as a distinguished research fellow and strategist for immune oncology discovery at AbbVie. Prior to this, he developed and led a genetic research institute at Sidra Medical and Research Center in Qata where he played a pivotal role in the Qatar Genome Project. He also trained in surgical oncology under Steven Rosenberg, M.D., Ph.D., at the National Cancer Institute and subsequently was a tenured investigator and chief of the infectious disease and immunogenetics section at the NIH Clinical Center. Dr. Marincola has spent his career studying tumor immunology and was a pioneer in the development of technologies for studying in real-time the dynamics of the tumor microenvironment adaptations during immune therapy. He described the mechanisms leading to cancer immune rejection describing the immunologic constant of rejection as a conserved process shared responsible for other forms of immune-mediated tissue-specific destruction such as allograft rejection, graft versus host disease, flares of autoimmunity and clearance of pathogen during acute infections. He is currently leading worldwide efforts to understand the mechanism of cancer immune resistance such as the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Task Force on Immune Responsiveness aimed at involving different areas of expertise besides immunology. Dr. Marincola graduated summa cum laude at the University of Milan, Medical School, Italy, and completed a general surgery residency with a focus in immunology at Stanford University. He was president of the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) and is the founding and current editor-in-chief of the Journal of Translational Medicine.

On May 2, 2018 Ziopharm Oncology, Inc. (Nasdaq:ZIOP), reported that management will host a conference call and webcast slide presentation on Thursday, May 10, at 4:30 p.m. ET to provide a corporate update and discuss financial results for the first quarter ended March 31, 2018 (Press release, Ziopharm, MAY 2, 2018, View Source [SID1234525979]).

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The call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 4857096. To access the slides and live webcast or the subsequent archived recording, visit the "Investors & Media" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.

On May 2, 2018 Triumvira Immunologics, a privately held biopharmaceutical company developing a novel platform for engineering T cells to attack multiple cancers, reported a strategic relationship with the Centre for Commercialisation of Cancer Immunotherapy (C3i), a catalyst for accelerating market access of breakthrough innovations to fight cancer (Press release, Triumvira Immunologics, MAY 2, 2018, View Source [SID1234525978]). Under the terms of the agreement, C3i will deliver cell therapy products for Triumvira’s global Phase 1 and 2 clinical trials and has committed to underwrite an investment in the company.

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"As we work toward our goal of entering clinical development in 2019, we understand the importance of securing sufficient product from a state-of-the-art facility."

"We are very pleased to be working with C3i for the manufacturing at the Centre of Excellence in Cellular Therapy (CETC) GMP manufacturing facility. It is critical for us to have a supplier with specific expertise in cellular therapies, including cancer immunotherapies," said Paul Lammers, MD, MSc, President & CEO, Triumvira. "As we work toward our goal of entering clinical development in 2019, we understand the importance of securing sufficient product from a state-of-the-art facility."

Francois Bettez, President & CEO of C3i, commented, "We are honored to be supporting Triumvira Immunologics in their drug development program. Their selective and targeted approach to treat cancer aligns well with our manufacturing expertise as well as our commitment to bringing new treatment options to patients with cancer."

As part of the deal, C3i also obtained a license to commercialize Triumvira’s lead product candidate, CD19 TAC, in Canada. "As part of the C3i mission, we not only build long-term partnerships but also ensure that the Canadians will have access to innovative technologies" commented Louisa Petropoulos, Director of Business Development at C3i.

The Centre of Excellence for Cellular Therapy (CETC), located at Hôpital Maisonneuve-Rosemont in Montreal, is a fully operational state-of-the art cGMP manufacturing facility for cellular therapies, including cancer immunotherapies and regenerative medicine. It is the only operational GMP validated center in Canada with commercial capacity, and is compliant with EMA, FDA and Health Canada regulatory requirements

About the Centre for Commercialisation of Cancer Immunotherapy
Established in 2016, the Centre for Commercialisation of Cancer Immunotherapy (C3i) is a Centre of Excellence for Commercialisation and Research from the Networks of Centres of Excellence. The mission is to accelerate access to innovative cancer immunotherapies for patients and offers an integrated structure for the development, translation and commercialization of groundbreaking therapies. C3i, which operates out of the Hôpital Maisonneuve- Rosemont in Montreal, Canada, combines four interacting units: GMP Manufacturing Unit for Regenerative Medicine and Cancer Immunotherapy; Biomarker-Diagnostic Unit; Clinical Research Unit; and Innovation and Commercialization Unit. For more information, visit: www.centrec3i.com

About Maisonneuve Rosemont Hospital (CIUSSS-EMTL)
Affiliated to the Université de Montréal, Hôpital Maisonneuve-Rosemont (HMR) is part of CIUSSS-EMTL. The HMR has a major research center of close to sixty researchers. There are four distinct sectors at the national and international levels: immuno-oncology, vision health, nephrology and cell therapy. Each year, the HMR receives more than 4,000 students, future physicians, nurses and health care professionals. HMR’s cancer program supports one of the largest bone marrow transplantation unit in Canada and is recognized a Canadian leader in cell therapy.